Management of Sickle Cell Disease: Present and Future

While the morbidity and mortality of sickle cell disease (SCD) has decreased in developed countries, the effects of sustained hemolytic anemia and episodic vaso-occlusive events often lead to end-organ complications and failure. In addition to chronic pain, progressive disease of the cardiopulmonary system, central nervous system, and kidneys have the most significant effects on patient morbidity and premature mortality.

Dr. Mark Gladwin will discuss the development of pulmonary hypertension, left ventricular diastolic heart disease, dysrhythmia, and sudden death in aging patients with SCD. He will also review the current status of screening and novel therapeutic approaches.

Dr. Elliott Vichinsky will summarize the epidemiology and mechanisms leading to progressive cognitive dysfunction and chronic kidney disease in adults with SCD. He will also provide a summary of the relationship between cerebrovascular infarction and regional atrophy with neurocognitive function. Dr. Vichinsky will review clinically important risk factors and biomarkers of neurocognitive decline and renal disease as well as therapeutic approaches using oxygen supplementation, hydroxyurea, transfusions, revascularization, and stem cell transplantation. Novel approaches for prevention of renal disease will be discussed, including angiotensin converting enzyme inhibitors, enothelin receptor blockade, and haptoglobin infusions.

Dr. Angela Rivers will review new therapeutic approaches based on the evolving role of intravascular hemolysis in SCD pathophysiology, including epigenetic induction of fetal hemoglobin, alternate mechanisms of hydroxyurea, anti-polymerization agents, chronic exchange transfusion, selectin inhibition, and lentivirus and CRISPR-CAS9-based gene therapy approaches to induce fetal hemoglobin and correct the beta-S-globin point mutation.